Fibrocell Science Inc FCSC is leveraging its expertise with fibroblasts to target monogenic diseases as well as broader indications. Roth Capital Partners’ Joseph Pantginis resumed coverage of the company with a Buy rating and a $7 price target.
Fibrocell plans using gene therapy to engineer fibroblasts to treat monogenic diseases and broader indications. The technology can take advantage of the inherent properties of fibroblasts, while combing this with the power of gene therapy to treat significant unmet medical needs, analyst Joseph Pantginis said.
Fibrocell has a technology-development partnership with Intrexon Corp XON, surrounding lentiviral vectors as well as the RheoSwitch Therapeutic System [RTS].
Phase II Data In June
The company’s lead product, azficel-T, is in Phase II development for vocal cord scarring and chronic or severe dysphonia. Positive proof-of-concept data has already been recorded and a Phase II is expected to read out in June 2016. “With current treatments, including surgery, only addressing underlying symptoms, azficel-T has the potential to be the first therapeutic for the indication,” Pantginis wrote.
Treating RDEB
Fibrocell has the opportunity to be the first “real helping hand” for kids with recessive dystrophic epidermolysis bullosa [RDEB]. The company’s FCX-007 is to enter a Phase I/II for RDEB, which is a devastating and progressive disease, often leading to death.
“Treatments for the disease at this point are only palliative and represent a major/catastrophic cost with bandaging (dressing, creams, supplies) and antibiotics alone, exceeding $10,000 per month. This baseline cost does not even include other pain medications, hospitalizations and surgeries,” the analyst commented.
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